Orphan Drugs Market Size, Share, Growth, Trends, and Global Industry Analysis: By Drug class (Biologics and Small Molecules) By Disease Type (Oncological Diseases, Metabolic Diseases, Hematologic and Immunologic Diseases, Infectious Diseases, and Neurologic Diseases) By Distribution channel (Hospitals Pharmacies, Retail Pharmacies, Online stores) Region Forecast 2020-2031

Orphan Drugs Market size was valued at US$ 237.4 billion in 2024 and is expected to reach US$ 477.5 billion by 2031, growing at a significant CAGR of 10.5% from 2025-2031. Orphan drugs are developed to treat rare medical conditions affecting less than 1 in 2,000 populations. Orphan drugs are used to treat patients with chronic severe diseases for which no treatment, or at least adequate one, has so far been available such as Agammaglobulinemia, Good pasture syndrome, Granulomatosis with Polyangiitis (GPA formerly Wagener Granulomatosis), Leucocyte adhesion deficiency, Pediatric Burton Agammaglobulinemia, Pediatric service combined Immunodeficiency, Schnitzler Syndrome and others. The market is growing significantly due to the rise in patient population, favorable reimbursement policies in developed and developing countries, and an increase in R&D for the launch of newer therapeutics by the market players. Moreover, pharmaceutical companies can price orphan drugs at premium levels due to the small patient population and the specialized nature of rare diseases, creating a proficient revenue potential.

In addition, regulatory incentives like decreased regulatory fees and tax credits further propel companies to invest in orphan drug development. Additionally, the rise in research and development efforts focused on addressing unmet medical needs, collaborations between academic institutions and market players, and fundraising from public and private organizations are anticipated to propel the market over the forecast years. However, the high cost of treatment, the stringent regulatory framework for product approval, and high R&D expenditure remain substantial barriers that hamper the growth of the market. However, the key opportunities associated with these drugs are enhanced affordability and patient access to these critical treatments, geographical expansion by the market players, and government incentives for R&D activities, which create opportunities for the players in the market.

Key Developments

• In March 2023, ADX-2191 molecule developed by Aldeyra Therapeutics, Inc received First FDA-Approved Drug for Patients Suffering from Primary Vitreoretinal Lymphoma.
• In September 2023, Ojjaara (momelotinib) received FDA approval for the myelofibrosis patients with anaemia condition.